PE20221578A1 - Moleculas de acido nucleico interferente corto (sina, por sus siglas en ingles) modificadas y usos de estas - Google Patents

Moleculas de acido nucleico interferente corto (sina, por sus siglas en ingles) modificadas y usos de estas

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Publication number
PE20221578A1
PE20221578A1 PE2022001900A PE2022001900A PE20221578A1 PE 20221578 A1 PE20221578 A1 PE 20221578A1 PE 2022001900 A PE2022001900 A PE 2022001900A PE 2022001900 A PE2022001900 A PE 2022001900A PE 20221578 A1 PE20221578 A1 PE 20221578A1
Authority
PE
Peru
Prior art keywords
nucleotide
nucleotides
sina
modified
fluoro
Prior art date
Application number
PE2022001900A
Other languages
English (en)
Spanish (es)
Inventor
Leonid Beigelman
Vivek Kumar Rajwanshi
Markus Hossbach
Rajendra K Pandey
Jin Hong
Laxman Eltepu
Saul Martinez Montero
Costa N Tilani S De
Original Assignee
Aligos Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Aligos Therapeutics Inc filed Critical Aligos Therapeutics Inc
Publication of PE20221578A1 publication Critical patent/PE20221578A1/es

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1131—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/20—Antivirals for DNA viruses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Cosmetics (AREA)
PE2022001900A 2020-03-06 2021-03-05 Moleculas de acido nucleico interferente corto (sina, por sus siglas en ingles) modificadas y usos de estas PE20221578A1 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062986150P 2020-03-06 2020-03-06
US202063109196P 2020-11-03 2020-11-03
PCT/US2021/021199 WO2021178885A1 (en) 2020-03-06 2021-03-05 Modified short interfering nucleic acid (sina) molecules and uses thereof

Publications (1)

Publication Number Publication Date
PE20221578A1 true PE20221578A1 (es) 2022-10-06

Family

ID=75278356

Family Applications (1)

Application Number Title Priority Date Filing Date
PE2022001900A PE20221578A1 (es) 2020-03-06 2021-03-05 Moleculas de acido nucleico interferente corto (sina, por sus siglas en ingles) modificadas y usos de estas

Country Status (15)

Country Link
US (4) US20220364096A1 (ko)
EP (1) EP4121533A1 (ko)
JP (1) JP2023515664A (ko)
KR (1) KR20220163960A (ko)
CN (1) CN115485384A (ko)
AU (1) AU2021230601A1 (ko)
BR (1) BR112022017287A2 (ko)
CA (1) CA3170466A1 (ko)
CL (1) CL2022002379A1 (ko)
CO (1) CO2022014258A2 (ko)
IL (1) IL296045A (ko)
MX (1) MX2022011012A (ko)
PE (1) PE20221578A1 (ko)
TW (1) TW202208625A (ko)
WO (1) WO2021178885A1 (ko)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2018332216B2 (en) * 2017-09-14 2024-05-02 Janssen Biopharma, Inc. Galnac derivatives
CN115485384A (zh) * 2020-03-06 2022-12-16 阿利戈斯治疗公司 经修饰的短干扰核酸(siNA)分子和其用途
AU2021365812A1 (en) 2020-10-21 2023-05-11 Aligos Therapeutics, Inc. Bicyclic compounds
WO2022184852A1 (en) * 2021-03-03 2022-09-09 Silence Therapeutics Gmbh Conjugated nucleic acids comprising a phosphorodithioate for inhibiting gene expression in a cell
US11957683B2 (en) 2021-06-18 2024-04-16 Aligos Therapeutics, Inc. Bicyclic compounds
WO2023122317A2 (en) * 2021-12-23 2023-06-29 Aligos Therapeutics, Inc. SHORT INTERFERING NUCLEIC ACID (SINA) MOLECULES FOR TARGETING β-CATENIN AND USES THEREOF
US20230399645A1 (en) * 2022-05-16 2023-12-14 University Of Massachusetts Optimized sirna scaffolds
WO2024033374A1 (en) 2022-08-11 2024-02-15 Syngenta Crop Protection Ag Novel arylcarboxamide or arylthioamide compounds
WO2024089216A1 (en) 2022-10-27 2024-05-02 Syngenta Crop Protection Ag Novel sulfur-containing heteroaryl carboxamide compounds
WO2024126650A1 (en) 2022-12-15 2024-06-20 Syngenta Crop Protection Ag Novel bicyclic-carboxamide compounds useful as pesticides
CN117534717A (zh) * 2024-01-09 2024-02-09 凯莱英生命科学技术(天津)有限公司 5′-(e)-乙烯基磷酸酯的合成方法

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110046206A1 (en) * 2007-06-22 2011-02-24 Isis Pharmaceuticals, Inc. Double strand compositions comprising differentially modified strands for use in gene modulation
EP2601204B1 (en) 2010-04-28 2016-09-07 Ionis Pharmaceuticals, Inc. Modified nucleosides and oligomeric compounds prepared therefrom
MX342731B (es) * 2011-06-30 2016-10-11 Arrowhead Res Corp Composiciones y metodos para inhibir la expresion genica del virus de hepatitis b.
CN104105790A (zh) * 2011-11-18 2014-10-15 阿尔尼拉姆医药品有限公司 修饰的RNAi试剂
WO2015125783A1 (ja) * 2014-02-18 2015-08-27 国立大学法人大阪大学 架橋型ヌクレオシドおよびヌクレオチド
KR20180038465A (ko) * 2015-08-07 2018-04-16 애로우헤드 파마슈티컬스 인코포레이티드 Ｂ형 간염 바이러스 감염에 대한 ｒｎａｉ 치료법
AU2017320582B2 (en) 2016-09-02 2023-11-16 Arrowhead Pharmaceuticals, Inc Targeting ligands
KR102493872B1 (ko) 2016-09-02 2023-01-30 다이서나 파마수이티컬, 인크. 4'-포스페이트 유사체 및 이를 포함하는 올리고뉴클레오타이드
EP4219713A3 (en) * 2017-04-05 2023-08-16 Silence Therapeutics GmbH Products and compositions
CA3083968C (en) * 2017-12-01 2024-04-23 Suzhou Ribo Life Science Co., Ltd. Double-stranded oligonucleotide, composition and conjugate comprising double-stranded oligonucleotide, preparation method thereof and use thereof
US20210238594A1 (en) * 2018-05-07 2021-08-05 Alnylam Pharmaceuticals, Inc. Compositions and methods for improving strand biased
MX2021005357A (es) 2018-11-08 2021-06-30 Aligos Therapeutics Inc Polimeros oligonucleotidos que inhiben el transporte de antigeno s y metodos.
CN115485384A (zh) * 2020-03-06 2022-12-16 阿利戈斯治疗公司 经修饰的短干扰核酸(siNA)分子和其用途

Also Published As

Publication number Publication date
TW202208625A (zh) 2022-03-01
CA3170466A1 (en) 2021-09-10
MX2022011012A (es) 2022-10-07
WO2021178885A1 (en) 2021-09-10
CL2022002379A1 (es) 2023-07-28
JP2023515664A (ja) 2023-04-13
US20220177888A1 (en) 2022-06-09
US20230332153A1 (en) 2023-10-19
EP4121533A1 (en) 2023-01-25
IL296045A (en) 2022-10-01
CN115485384A (zh) 2022-12-16
KR20220163960A (ko) 2022-12-12
US20230365970A1 (en) 2023-11-16
AU2021230601A2 (en) 2022-10-20
AU2021230601A1 (en) 2022-10-06
US20220364096A1 (en) 2022-11-17
US11549110B2 (en) 2023-01-10
CO2022014258A2 (es) 2022-12-30
BR112022017287A2 (pt) 2022-11-16

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